2023
Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States.
Goshua G, Calhoun C, Ito S, James L, Luviano A, Krishnamurti L, Pandya A. Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States. Annals Of Internal Medicine 2023, 176: 779-787. PMID: 37247420, PMCID: PMC10370480, DOI: 10.7326/m22-3272.Peer-Reviewed Original ResearchFemale Reproductive Health Outcomes after Hematopoietic Cell Transplantation for Sickle Cell Disease: Is Reduced Intensity Better Than Myeloablative Conditioning?
Meacham L, George S, Veludhandi A, Pruett M, Haight A, Arnold S, Elchuri S, Stenger E, Krishnamurti L. Female Reproductive Health Outcomes after Hematopoietic Cell Transplantation for Sickle Cell Disease: Is Reduced Intensity Better Than Myeloablative Conditioning? Transplantation And Cellular Therapy 2023, 29: 531.e1-531.e4. PMID: 37169288, DOI: 10.1016/j.jtct.2023.05.004.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationReduced-intensity conditioningPremature ovarian insufficiencySickle cell diseaseNormal AMH levelsMyeloablative conditioningAnti-Müllerian hormoneOvarian outcomeAMH levelsCell transplantationCell diseaseRIC HCTFollicle-stimulating hormone levelsPediatric oncology patientsRisk of infertilityMIU/mLStudy 2 patientsReproductive health outcomesMelphalan regimenConditioning regimenGonadal damageOvarian damageConditioning regimensOvarian reserveRIC regimensParental perspective on the risk of infertility and fertility preservation options for children and adolescents with sickle cell disease considering hematopoietic stem cell transplantation
Sinha C, Meacham L, Bakshi N, Ross D, Krishnamurti L. Parental perspective on the risk of infertility and fertility preservation options for children and adolescents with sickle cell disease considering hematopoietic stem cell transplantation. Pediatric Blood & Cancer 2023, 70: e30276. PMID: 37051746, PMCID: PMC10544372, DOI: 10.1002/pbc.30276.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationRisk of infertilitySickle cell diseaseStem cell transplantationFertility preservationCell transplantationCell diseaseMajor long-term complicationPrimary caregiversIdentical related donorsDisease-free survivalFertility preservation optionsFertility preservation proceduresLong-term complicationsHuman leukocyte antigenConditioning regimenHost diseaseRelated donorsLeukocyte antigenPreservation optionsHCT physiciansSurvival rateAvailable HLAEleven participantsInfertility
2022
Multimodal phenotyping and correlates of pain following hematopoietic cell transplant in children with sickle cell disease
Bakshi N, Astles R, Chou E, Hurreh A, Sil S, Sinha C, Sanders K, Peddineni M, Gillespie S, Keesari R, Krishnamurti L. Multimodal phenotyping and correlates of pain following hematopoietic cell transplant in children with sickle cell disease. Pediatric Blood & Cancer 2022, 70: e30046-e30046. PMID: 36322607, PMCID: PMC9820671, DOI: 10.1002/pbc.30046.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantPatient-reported outcomesSickle cell diseaseYear post-HCTPost-HCTExperimental pain sensitivitySickle cell anemiaCell transplantPain thresholdPain sensitivityCell diseaseCorrelates of painPressure pain thresholdHealth-related qualityCold pain thresholdAssessment of painPsychological factorsUnderstanding of painEffect sizePain assessmentObservational studySevere genotypePainCell anemiaOptional substudiesDecision-making about gene therapy in transfusion dependent thalassemia
Quarmyne M, Ross D, Sinha C, Bakshi N, Boudreaux J, Krishnamurti L. Decision-making about gene therapy in transfusion dependent thalassemia. BMC Pediatrics 2022, 22: 536. PMID: 36085025, PMCID: PMC9461218, DOI: 10.1186/s12887-022-03598-3.Peer-Reviewed Original ResearchConceptsTransfusion-dependent thalassemiaPatient/family knowledgeDependent thalassemiaTransfusion independenceTreatment optionsStudy participantsGene therapyFamily knowledgeElimination of transfusionsFrequency of transfusionMorbidity/mortalityStem cell transplantationLong-term outcomesPreferred treatment modalityPromising treatment optionBackgroundHematopoietic stem cell transplantationPatients/familiesMethodsParents of childrenCurative intentTransfusion reductionHost diseaseDonor HSCTParents of childrenCell transplantationMean ageAssociated comorbidities, healthcare utilization & mortality in hospitalized patients with haemophilia in the United States: Contemporary nationally representative estimates
Day J, Takemoto C, Sharathkumar A, Makhani S, Gupta A, Bitner S, Josephson C, Bloch E, Tobian A, Krishnamurti L, Goel R. Associated comorbidities, healthcare utilization & mortality in hospitalized patients with haemophilia in the United States: Contemporary nationally representative estimates. Haemophilia 2022, 28: 532-541. PMID: 35412659, PMCID: PMC9540439, DOI: 10.1111/hae.14557.Peer-Reviewed Original ResearchConceptsNationwide Inpatient SampleHealthcare utilizationPrevalence of comorbiditiesAge-related comorbiditiesCentral line infectionsInpatient discharge databaseOverall mortality rateCatheter-related infectionsHealthcare utilization patternsMedian hospital chargesICD-10 codesRepresentative estimatesCause admissionsCause hospitalizationAdult PWHAssociated comorbidityCommon comorbiditiesHospitalized patientsMedian ageContemporary cohortHospital burdenHospital chargesDischarge databaseInpatient SampleLine infections
2020
Mortality in sickle cell disease: A population‐based study in an aboriginal community in the Gudalur Valley, Nilgiris, Tamil Nadu, India
Sheshadri V, Shabeer P, Santhirapala V, Jayaram A, Krishnamurti L, Menon N. Mortality in sickle cell disease: A population‐based study in an aboriginal community in the Gudalur Valley, Nilgiris, Tamil Nadu, India. Pediatric Blood & Cancer 2020, 68: e28875. PMID: 33381914, DOI: 10.1002/pbc.28875.Peer-Reviewed Original ResearchConceptsSickle cell diseaseCause of deathCell diseaseMortality rateSeverity of SCDAge groupsAboriginal populationAcute chest syndromePopulation-based studyPercent of deathsCrude mortality rateCommunity-based comprehensive careCause of mortalityCases of deathPaucity of dataCommon monogenic disorderChest syndromeMedian ageHospital recordsSCD patientsAutopsy questionnaireAboriginal communitiesComprehensive careLongitudinal cohortRemote Aboriginal communitiesPrimary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease
Sinha C, Bakshi N, Ross D, Loewenstein G, Krishnamurti L. Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease. Pediatric Blood & Cancer 2020, 68: e28749-e28749. PMID: 33034129, PMCID: PMC8246626, DOI: 10.1002/pbc.28749.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseasePrimary caregiversSCD complicationsCell transplantationCell diseaseDiminished qualityAutologous hematopoietic progenitor cellsPrimary caregiver reportMajor medical decisionsGene therapyCurative optionSevere complicationsHematopoietic progenitor cellsClinical trialsAcceptable treatmentRecent complicationsComplicationsCaregiver reportsCaregiversProgenitor cellsNormal lifeTransplantationTherapyMedical decisionsPatient and family experience with chronic transfusion therapy for sickle cell disease: A qualitative study
Hawkins L, Sinha C, Ross D, Yee M, Quarmyne M, Krishnamurti L, Bakshi N. Patient and family experience with chronic transfusion therapy for sickle cell disease: A qualitative study. BMC Pediatrics 2020, 20: 172. PMID: 32305060, PMCID: PMC7165370, DOI: 10.1186/s12887-020-02078-w.Peer-Reviewed Original ResearchConceptsChronic transfusion therapySickle cell diseaseTransfusion therapyCell diseaseHealthcare providersPrevention of complicationsFamily experiencesStroke preventionVenous accessPatient knowledgeChildren 12Future studiesPatient participantsChelation therapySignificant patientFamily burdenSubstantial burdenPatient experiencePatientsSemi-structured interview formatTherapyComplicationsResultsFour themesBurdenInformed decision-making process
2019
How I treat sickle cell disease with hematopoietic cell transplantation
Stenger E, Shenoy S, Krishnamurti L. How I treat sickle cell disease with hematopoietic cell transplantation. Blood 2019, 134: 2249-2260. PMID: 31697818, PMCID: PMC6923666, DOI: 10.1182/blood.2019000821.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseCell transplantationCell diseaseOrgan dysfunctionSCD patientsSignificant morbidityEarly mortalityClinical trialsAvailable cureSCD recipientsAlternative donorsIndividual patientsClinical practicePatientsTransplantationClinical vignettesDiseaseOutcomesMorbidityDysfunctionMortalityTrialsRecipientsComparative Effectiveness of a Web-Based Patient Decision Aid for Therapeutic Options for Sickle Cell Disease: Randomized Controlled Trial
Krishnamurti L, Ross D, Sinha C, Leong T, Bakshi N, Mittal N, Veludhandi D, Pham A, Taneja A, Gupta K, Nwanze J, Matthews A, Joshi S, Olivieri V, Arjunan S, Okonkwo I, Lukombo I, Lane P, Bakshi N, Loewenstein G. Comparative Effectiveness of a Web-Based Patient Decision Aid for Therapeutic Options for Sickle Cell Disease: Randomized Controlled Trial. Journal Of Medical Internet Research 2019, 21: e14462. PMID: 31799940, PMCID: PMC6934048, DOI: 10.2196/14462.Peer-Reviewed Original ResearchConceptsRandomized clinical trialsPatient decision aidSickle cell diseaseWeb-based patient decision aidClinical trialsHealth care providersCell diseaseDecisional conflictPatient knowledgeCare providersDecision aid armStandard care armOttawa Decision Support FrameworkChronic blood transfusionsDisease-modifying therapiesBone marrow transplantationDecision aidCare armDecision aid prototypeClinical characteristicsBlood transfusionControlled TrialsPediatric patientsMarrow transplantationTherapeutic options
2018
Ovarian Sertoli–Leydig tumor after bone marrow transplant for sickle cell disease
Phillips L, Krishnamurti L, Rytting H, Olson T. Ovarian Sertoli–Leydig tumor after bone marrow transplant for sickle cell disease. Pediatric Blood & Cancer 2018, 65: e27367. PMID: 30039911, DOI: 10.1002/pbc.27367.Peer-Reviewed Original Research
2017
Experiences and Decision Making in Hematopoietic Stem Cell Transplant in Sickle Cell Disease: Patients' and Caregivers' Perspectives
Khemani K, Ross D, Sinha C, Haight A, Bakshi N, Krishnamurti L. Experiences and Decision Making in Hematopoietic Stem Cell Transplant in Sickle Cell Disease: Patients' and Caregivers' Perspectives. Transplantation And Cellular Therapy 2017, 24: 1041-1048. PMID: 29196076, DOI: 10.1016/j.bbmt.2017.11.018.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantSickle cell diseaseStem cell transplantPatient-caregiver dyadsCell transplantCell diseaseSuccessful hematopoietic stem cell transplantAdult long-term survivorsTransplant-related morbidityDisease-related complicationsLong-term survivorsQuality of lifeSemistructured interview guideFocus group sessionsCurative intentHSCT decisionChildhood morbidityCaregivers' knowledgeHSCT experiencePremature mortalityDaily livingDecisional regretPatientsCaregiver perspectivesChild healthcareProponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease
Bakshi N, Sinha C, Ross D, Khemani K, Loewenstein G, Krishnamurti L. Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease. PLOS ONE 2017, 12: e0178413. PMID: 28727801, PMCID: PMC5518995, DOI: 10.1371/journal.pone.0178413.Peer-Reviewed Original ResearchConceptsSickle cell diseaseChronic blood transfusionsBone marrow transplantationDisease-modifying therapiesTreatment-related decisionsTreatment optionsCell diseasePhysician perspectivesAvailable disease-modifying therapiesLong-term adverse effectsPhysician-related factorsAvailable treatment optionsDisease-related factorsPossible treatment optionsPatients/familiesInherited blood disorderBlood transfusionMarrow transplantationPhysician interviewsPatient burdenPhysicians' perceptionsPatient's perspectivePhysicians' approachPatient engagementTreatment planDetermining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module
Panepinto J, Paul Scott J, Badaki-Makun O, Darbari D, Chumpitazi C, Airewele G, Ellison A, Smith-Whitley K, Mahajan P, Sarnaik S, Charles Casper T, Cook L, Leonard J, Hulbert M, Powell E, Liem R, Hickey R, Krishnamurti L, Hillery C, Brousseau D, for the Pediatric Emergency Care Applied Research Network (PECARN). Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module. Health And Quality Of Life Outcomes 2017, 15: 124. PMID: 28606098, PMCID: PMC5468970, DOI: 10.1186/s12955-017-0700-2.Peer-Reviewed Original ResearchConceptsSickle Cell Disease ModuleDisease-specific HRQL instrumentsHealth-related qualityPatient-centered outcomesProspective trial designSickle cell diseaseSpecific HRQL instrumentsHRQL assessmentHRQL instrumentsLongitudinal validityAncillary studiesCell diseaseTrial designHealth statusDisease modulesTime pointsPedsQLMeaningful changeChildrenHRQLPatientsHospitalDiseasePsychological Characteristics and Pain Frequency Are Associated With Experimental Pain Sensitivity in Pediatric Patients With Sickle Cell Disease
Bakshi N, Lukombo I, Shnol H, Belfer I, Krishnamurti L. Psychological Characteristics and Pain Frequency Are Associated With Experimental Pain Sensitivity in Pediatric Patients With Sickle Cell Disease. Journal Of Pain 2017, 18: 1216-1228. PMID: 28602692, DOI: 10.1016/j.jpain.2017.05.005.Peer-Reviewed Original ResearchConceptsSickle cell diseaseExperimental pain stimuliExperimental pain sensitivityPain sensitivityPain stimuliChronic painExperimental painCell diseasePsychological characteristicsQuantitative sensory testing methodsPsychological factorsChronic SCD painMechanical temporal summationVaso-occlusive painQuantitative sensory testingSubset of patientsPain-related outcomesStudy of patientsCross-sectional assessmentChildren ages 8SCD painPain burdenPain frequencyPediatric patientsPain processingNovel Metrics in the Longitudinal Evaluation of Pain Data in Sickle Cell Disease
Bakshi N, Smith M, Ross D, Krishnamurti L. Novel Metrics in the Longitudinal Evaluation of Pain Data in Sickle Cell Disease. The Clinical Journal Of Pain 2017, 33: 517-527. PMID: 27584817, DOI: 10.1097/ajp.0000000000000431.Peer-Reviewed Original ResearchConceptsMaximum daily painSickle cell diseasePain intensity dataChronic painCell diseasePain dataInterindividual variationMaximum daily pain scoresPatient-reported end pointsDaily pain scoresPain-free daysInterventional clinical trialsBurden of painPeriod of hospitalizationElectronic pain diaryDiary daysHealth care providersPain burdenPain scoresPain diaryPain intensityDaily painSCD patientsPain trendsClinical trialsBone Marrow–Derived Mesenchymal Stromal Cells from Patients with Sickle Cell Disease Display Intact Functionality
Stenger E, Chinnadurai R, Yuan S, Garcia M, Arafat D, Gibson G, Krishnamurti L, Galipeau J. Bone Marrow–Derived Mesenchymal Stromal Cells from Patients with Sickle Cell Disease Display Intact Functionality. Transplantation And Cellular Therapy 2017, 23: 736-745. PMID: 28132869, PMCID: PMC5390328, DOI: 10.1016/j.bbmt.2017.01.081.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAnemia, Sickle CellCell CommunicationCell Culture TechniquesCell ProliferationChildChild, PreschoolFemaleHealthy VolunteersHematopoietic Stem Cell TransplantationHumansImmunophenotypingIndoleamine-Pyrrole 2,3,-DioxygenaseMaleMesenchymal Stem Cell TransplantationMesenchymal Stem CellsTransplantation, AutologousYoung AdultConceptsSickle cell diseaseHematopoietic cell transplantationMesenchymal stromal cellsAutologous mesenchymal stromal cellsBone marrowTime of HCTAutologous T cell proliferationStromal cellsThird-party mesenchymal stromal cellsBM-derived mesenchymal stromal cellsMajor histocompatibility complex compatibilityExperimental murine modelT cell proliferationSurface marker phenotypeDose-dependent mannerIFN-γ stimulationAmeliorate graftHost diseaseSCD patientsCell transplantationImmunomodulatory pathwaysSCD subjectsCell diseaseHealthy volunteersMurine model
2016
Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation
Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simões B, Ferster A, Dupont S, de la Fuente J, Dalle J, Zecca M, Walters M, Krishnamurti L, Bhatia M, Leung K, Yanik G, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier J, Ayas M, Cavazzana M, Matthes-Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood 2016, 129: 1548-1556. PMID: 27965196, PMCID: PMC5356458, DOI: 10.1182/blood-2016-10-745711.Peer-Reviewed Original ResearchConceptsEvent-free survivalSickle cell diseaseGraft failureCell transplantationHLA-identical sibling transplantationReduced-intensity conditioning regimensHLA-identical sibling transplantsHematopoietic stem cell transplantationMyeloablative conditioning regimenHematopoietic cell transplantationStem cell transplantationCox regression modelMarrow Transplant ResearchBenefit of transplantStem cell sourceSibling transplantationConditioning regimenPrimary endpointConditioning regimensMost patientsOverall survivalSibling transplantsCurative therapyInternational BloodMedian ageA trial of unrelated donor marrow transplantation for children with severe sickle cell disease
Shenoy S, Eapen M, Panepinto J, Logan B, Wu J, Abraham A, Brochstein J, Chaudhury S, Godder K, Haight A, Kasow K, Leung K, Andreansky M, Bhatia M, Dalal J, Haines H, Jaroscak J, Lazarus H, Levine J, Krishnamurti L, Margolis D, Megason G, Yu L, Pulsipher M, Gersten I, DiFronzo N, Horowitz M, Walters M, Kamani N. A trial of unrelated donor marrow transplantation for children with severe sickle cell disease. Blood 2016, 128: 2561-2567. PMID: 27625358, PMCID: PMC5123194, DOI: 10.1182/blood-2016-05-715870.Peer-Reviewed Original ResearchConceptsEvent-free survivalIncidence rateAcute chest syndrome episodesPosterior reversible encephalopathy syndromeSevere sickle cell diseaseUnrelated donor marrow transplantationVaso-occlusive pain crisesAllogeneic bone marrow transplantEffective GVHD prophylaxisGVHD-related deathsHost disease (GVHD) prophylaxisShort-course methotrexateReversible encephalopathy syndromeUnrelated donor transplantsPhase 2 trialBone marrow transplantTranscranial Doppler velocitiesSickle cell diseaseAcute GVHDChronic GVHDEFS ratesEncephalopathy syndromeGVHD prophylaxisTransplant indicationConditioning regimen